Ocular Melanoma Foundation

Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of November 13, 2017

Epizyme released results of a phase I trial of tazemetostat in pediatric patients with relapsed or refractory INI1-negative molecularly defined solid tumors. The open-label, multi-dose, multicenter dose escalation study was conducted in 46 patients aged 6 months to 21 years with INI1-negative tumors including epithelioid sarcoma, poorly differentiated chordoma, atypical teratoid rhabdoid tumors, malignant rhabdoid tumors, renal medullary carcinoma or relapsed/refractory synovial sarcoma. The oral suspension of tazemetostat was administered twice daily in continuous 28-day cycles in the following cohorts: 240mg/m2, 300mg/m2, 400mg/m2, 520mg/m2, 700mg/m2, 900mg/m2, 1200mg/m2. Tazemetostat was generally well-tolerated at all explored doses, including the highest dose tested. Adverse events (AEs) reported, regardless of attribution, were mostly mild to moderate, the most common of which were vomiting (41%), pyrexia (28%), headache (24%) and nausea (24%). Only one patient experienced a dose-limiting toxicity (DLT) event at the dose level of 300mg/m2 (grade four dyspnea and grade three hypoxia); however, no other DLTs were observed at higher doses. One other patient discontinued the study due to a treatment-related AE, and five patients had dose reductions. Tazemetostat showed encouraging anti-tumor activity across a range of INI1-negative cancers in pediatric patients. Complete or partial responses were observed in patients at dose levels ranging from 520 to 900 mg/m2 twice daily, as follows: complete responses in epithelioid sarcoma (n=1), chordoma (n=1), atypical teratoid rhabdoid tumor (n=1); partial response in chordoma (n=1). The recommended phase II dose of 1200 mg/m2 twice daily was established based on safety, pharmacokinetics, pharmacodynamics and activity. The study is now enrolling patients into four dose expansion cohorts.

Millendo Therapeutics reported announced positive topline results from a phase II proof-of-concept study of ATR-101 in classic congenital adrenal hyperplasia (CAH). The trial was a multicenter, single-blind, multiple dose study that assessed the efficacy and safety of orally-administered ATR-101 in addition to corticosteroids in patients with classic congenital adrenal hyperplasia resulting from 21-hydroxylase deficiency. The study, which assessed five escalating doses, alternated between two weeks of treatment with ATR-101 and two weeks with placebo to determine the effects of ATR-101 on adrenal steroids. In the study, seven of the first 10 patients demonstrated a clear biological effect as measured by reductions in 17-hydroxyprogesterone (17-OHP), a key measure of disease control. Based on the trial results, Millendo ended the trial early and the FDA has granted orphan drug status to ATR-101 in this indication. Mean reductions in 17-OHP were observed at all ATR-101 doses while mean increases were observed during all placebo treatments. Two patients experienced a reduction in 17-OHP levels to ≤2x ULN, the primary endpoint, a result consistent with the short duration of treatment (two weeks/dose level) and high baseline levels. ATR-101 was well-tolerated at all dose levels. Separately, Millendo has decided to discontinue development of MLE4901 after assessment of the clinical risks and benefits of the program.

Pique Therapeutics announced positive results for PT 107 from its phase II clinical trial in patients with late-stage, second-line non-small cell lung cancer (NSCLC). The trial was multicenter, randomized (2:1), double-blind and enrolled 94 patients. Median overall survival for patients receiving treatment with PT 107 was 12.5 months versus 8.4 months for patients who received placebo, with a hazard ratio of 0.65 (p=0.06, all p-values two-sided). In the subset of 71 non-squamous patients, median overall survival was 15.5 months (PT 107 arm) vs. 5.8 months (control arm), with a hazard ratio of 0.46 (p<0.01). Median time to progression for the PT 107 arm was 4.0 months versus 2.5 months for the control arm, with a statistically significant hazard ratio of 0.45 (p=0.01). Median progression-free survival for the PT 107 arm was 3.9 months versus 2.9 months for the control arm, with a hazard ratio of 0.67 (p=0.08). There were no drug-related serious adverse events reported for patients on PT 107, consistent with previous clinical experience, and minimal causal grade 3+ toxicity (5%).